Clinical trial
A study to evaluate the safety, efficacy, PK, PD and immunogenicity of cipaglucosidase alfa/miglustat in IOPD subjects aged 0 to <18 (ROSSELLA)
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 (ROSSELLA)
ClinicalTrials.gov ID: NCT04808505
Sponsor: Amicus Therapeutics
Information provided by: Amicus Therapeutics (Responsible Party)
Last Update Posted: 2024-09-19
Brief Summary:
This is a Phase 3, open-label, multicenter study to evaluate the safety, efficacy, PK, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-naïve pediatric subjects with IOPD.
Official Title:
An Open-label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Both ERT-experienced and ERT-naïve Pediatric Subjects With Infantile-onset Pompe Disease Aged 0 to < 18 Years
Intervention / Treatment:
- Biological: Cipaglucosidase alfa
- Drug: Miglustat
| Category | Value |
|---|---|
| Study Start (Actual) |
2023-07-18
|
| Primary Completion (Estimated) |
2027-04-01
|
| Study Completion (Estimated) |
2027-04-01
|
| Enrollment (Estimated) | 36 |
| Study Type | Interventional |
| Phase | Phase 3 |
| Other Study ID Numbers |
ATB200-08
|